FDA Approves Drug That Could Treat 90% of Cystic Fibrosis Patients

More than 30,000 people in the United States suffer from the life-threatening disease

Published October 23, 2019•Updated on October 23, 2019 at 3:30 pm

Business Wire

The U.S. Food and Drug Administration announced Wednesday that it has approved the first drug combination to treat most cystic fibrosis patients.

Boston-based company Vertex Pharmaceuticals Inc. created Trikafta, which combined three drugs in one. Trikafta has been approved for those aged 12 and older with at least one F508del mutation, which affects 90%, or 27,000 patients, with the lung disease in the United States.

It’s also the first drug that will target the direct cause of the disease for 6,000 patients in the United States, according to Vertex.

The FDA said in a statement that it has consistently aimed to increase the development of new drug therapies like Trikafta while standing by its review standards.

“Today’s landmark approval is a testament to these efforts, making a novel treatment available to most cystic fibrosis patients, including adolescents, who previously had no options and giving others in the cystic fibrosis community access to an additional effective therapy,” it said.

More than 30,000 people in the United States suffer from the life-threatening disease, according to the Cystic Fibrosis Foundation Patient Registry. Cystic fibrosis causes a build-up of mucus in the lungs, digestive system and in other parts of the body, leading to severe respiratory and digestive complications, according to the FDA.

The lung disease is linked to mutations in the CFTR gene, with the most common being the F508del mutation. Trikafta helps the protein created by the CFTR gene work more effectively. Previous drugs created to target the protein failed to help patients with other kinds of mutations in the CFTR gene, according to the FDA.

In two separate studies, Vertex Pharmaceuticals Inc. found that Trikafta alleviated respiratory and digestive symptoms associated with cystic fibrosis.

"If you think about it, we went from being able to treat no patients to being able to treat nearly all patients in seven years," Vertex Pharmaceuticals CEO Jeff Leiden told the Boston Business Journal.